uniQure announces Phase I/II study of AMT-130 met primary endpoint

uniQure (QURE) N.V. announced positive topline data from the pivotal Phase I/II study of AMT-130 for the treatment of Huntington’s disease. The study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale at 36 months compared to a propensity score-matched external control. The study also met a key secondary endpoint by achieving statistically significant slowing of disease progression as measured by Total Functional Capacity at 36 months compared to a propensity score-matched external control. Topline 36-month efficacy results for patients receiving high-dose AMT-130 are as follows : A statistically significant 75% slowing of disease progression as measured by cUHDRS, which met the primary endpoint of the study. Treated patients had a mean change in cUHDRS from baseline of -0.38 compared to a change of -1.52 for patients in the propensity score-matched external control. A statistically significant 60% slowing of disease progression as measured by TFC, which met a key secondary endpoint of the study. Favorable trends in other secondary endpoint measures of motor and cognitive function. A mean reduction from baseline in cerebrospinal neurofilament light protein of -8.2%.